Ban Hong-Seok, Lee Sang-Kyung K, Kumar Priti
Hanyang University, Department of Bioengineering, 17 Haengdang-dong Seongdong-gu, Seoul 133-797, South Korea.
IDrugs. 2009 Dec;12(12):774-8.
The ability to block the expression of any disease-causing gene or disease-related protein highlights the potential use of RNAi technology in the therapy of 'undruggable' human diseases. However, considering the risks associated with RNAi therapy, targeting and restricting the action of siRNA to specific cells could greatly minimize toxic side effects. However, this is a major challenge, as many primary cell types are highly recalcitrant to siRNA uptake. This review discusses advances in siRNA targeting methods for human T-cells, with an emphasis on the potential use of an RNAi-based therapy for the treatment of HIV/AIDS.
阻断任何致病基因或疾病相关蛋白表达的能力凸显了RNA干扰(RNAi)技术在治疗“不可成药”人类疾病方面的潜在用途。然而,考虑到RNAi治疗相关的风险,将小干扰RNA(siRNA)的作用靶向并限制于特定细胞可极大地减少毒副作用。然而,这是一项重大挑战,因为许多原代细胞类型对siRNA摄取具有高度抗性。本综述讨论了针对人类T细胞的siRNA靶向方法的进展,重点是基于RNAi的疗法在治疗HIV/AIDS方面的潜在用途。